Severe Hemophilia A
Conditions
Keywords
Pharmacokinetics;, Safety;, immunogenicity;, Preliminary efficacy
Brief summary
Primary objective: To assess the pharmacokinetics, Safety and immunogenicity of Repeat Dosing of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection With Severe Hemophilia A(FRSW117) Secondary objectives: To assess Preliminary efficacy of Repeat Dosing of PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection With Severe Hemophilia A.
Interventions
DRUGFRSW117
Subjects of high dose group are being received four doses(50 IU/kg,once a week or every 7 days) of FRSW117. Subjects of low dose group (40 IU/kg,once a week or every 7 days)are being received four doses of FRSW117.
Sponsors
Jiangsu Gensciences lnc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
12 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * The activity of the coagulation factor VIII (FVIII:C) \< 1%. * Patients previously treated with FVIII concentrate (s) for a minimum of 150 exposure days (EDs) prior to study entry * Normal prothrombin time or INR \< 1.3 * Negative lupus anticoagulant Key
Exclusion criteria
* Hypersensitive to any of the excipients of the test materials (e.g. allergic to murine or hamster origin heterologous proteins) * History of hypersensitivity or anaphylaxis associated with any FVIII or II immunoglobulin administration * Current FVIII inhibitor-positive or history of FVIII inhibitor-positive * Other coagulation disorder(s) in addition to hemophilia A.• Significant hepatic or renal impairment (ALT and AST \> 2×ULN; serum bilirubin level \> 2 × upper limit of normal (ULN), Urea /BUN \> 2×ULN, Cr \> 176.8 µmol/L) * One or more clinically significant tests for Human Immunodeficiency Virus (HIV), Antisyphilitic spirulina (TPHA) and Hepatitis C Virus (HCV) Antibody * Patients who received any anticoagulant or antiplatelet therapy within one week prior screening or need to receive an anticoagulant or antiplatelet therapy during the period of clinical trials * Patients having major surgery or receiving blood or bood components transfusion within 4 weeks prior screening or having planned major surgery schedule during the study * Patients who previously participated in the other clinical trials within one month prior screening * Any life-threatening disease or condition which, according to the investigator's judgment, could not benefit from the trial participation * Patient who is considered by the other investigators not suitable for clinical study Other protocol-defined inclusion/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Evaluation of the level of anti-PEG antibody production in participants | Pre-dose and post dose up to 36 days | — |
| Area Under the Curve to Infinity (AUC) | Pre-dose and post dose up to 10 days | Measured by One-Stage Clotting Assay |
| The measure of the efficiency of the body to remove the drug and the unit is the volume of the plasma or blood cleared of drug per unit time (CL) | Pre-dose and post dose up to 10 days | Measured by One-Stage Clotting Assay |
| Evaluation of the level of anti-PEG-rFⅧFc antibody production in participants | Pre-dose and post dose up to 36 days | — |
| Maximum measured concentration of FVIII:C (Cmax) | Pre-dose and post dose up to 10 days | Measured by One-Stage Clotting Assay |
| Time required for the concentration of the drug to reach half of its original value (T1/2) | Pre-dose and post dose up to 10 days | Measured by One-Stage Clotting Assay |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Development of Inhibitor | Pre-dose and post dose up to 36 days | Measured by the Nijmegen-Modified Bethesda Assay |
| score of bleeding symptoms and Vital signs | Pre-dose and post dose up to 36 days | Response to treatment with PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for bleeding episodes, using the 4-point bleeding response scale |
| Number of participants with treatment-related adverse events as assessed by CTCAE V5.0 | Pre-dose and post dose up to 36 days | Adverse events related to PEG Recombinant Human Coagulation Factor VIII-Fc Fusion Protein according to Common Terminology Criteria for Adverse Events (CTCAE) NCI.V5.0. |
Countries
China
Outcome results
None listed